Benitec Biopharma reported that the first patient treated in Cohort 1 of its Phase 1b/2a study for oculopharyngeal muscular dystrophy (OPMD) has completed a 24‑month post‑treatment assessment, showing continued improvement in swallowing function and a marked reduction in dysphagic symptom burden compared with the 12‑month timepoint. The data include a 27 % increase in the PhAMPC score, a 60 % decrease in the NRRSv score, a 39 % drop in the TPR score, and a 78 % reduction in the SSQ score, confirming that the therapeutic effect is sustained over two years.
All four patients in Cohort 1 met the pre‑specified responder criteria at the 12‑month follow‑up, giving the study a 100 % response rate. The uniform response across multiple clinical endpoints underscores the consistency of the gene‑therapy platform and strengthens the evidence base that BB‑301 can deliver durable, disease‑modifying benefits in OPMD.
BB‑301 operates on Benitec’s “Silence and Replace” DNA‑directed RNA interference platform, which simultaneously silences the mutant PABPN1 gene responsible for OPMD and delivers a functional replacement gene. The single‑administration therapy permanently suppresses the pathogenic allele while providing a sustained source of normal protein, a strategy that has not yet been demonstrated in any other OPMD treatment.
The durable efficacy data reinforce Benitec’s regulatory strategy. The company has already secured Orphan Drug and Fast Track designations for BB‑301, and it has scheduled a meeting with the FDA in mid‑2026 to discuss the design of a pivotal study. The meeting will focus on confirming the long‑term benefit profile and defining the endpoints that will support a regulatory submission.
Benitec’s clinical progress is supported by a strong financial foundation. The company raised $100 million in a capital‑raising round in November 2025, providing a cash runway that enables continued development of BB‑301 and other pipeline assets. Safety data from the 24‑month assessment were reassuring, with no new safety signals reported. Management emphasized that the durable improvements in dysphagic symptom burden could translate into meaningful quality‑of‑life gains for patients, and the company remains focused on advancing BB‑301 through development to improve the lives of all OPMD patients.
The 24‑month efficacy announcement marks a pivotal milestone for Benitec Biopharma, confirming that BB‑301 delivers sustained, clinically meaningful benefits in OPMD. The data support the company’s regulatory strategy, strengthen its commercial case, and reinforce confidence in the underlying platform and the company’s ability to secure the necessary funding to bring the therapy to market.
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